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To Determine The Efficacy and Safety of GDC-0449 in Patients With Basal Cell Nevus Syndrome (BCNS) (GDC-0449)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00957229
Recruitment Status : Completed
First Posted : August 12, 2009
Last Update Posted : June 6, 2016
Genentech, Inc.
Information provided by (Responsible Party):
UCSF Benioff Children's Hospital Oakland

Brief Summary:
The purpose of this study is to reduce the number of new surgically eligible BCCs by 50% appearing during month 3-18 of medication ingestion.

Condition or disease Intervention/treatment Phase
Basal Cell Nevus Syndrome Gorlin Syndrome Drug: GDC-0449 Phase 2

Detailed Description:
This is a Phase II, 18 month, double blind, randomized placebo-controlled, two arm multicenter clinical study design. During the 18-month treatment period, the safety and chemopreventive efficacy of 150 mg/day GDC-0449 versus placebo will be assessed, and include evaluations of the skin at monthly intervals for the first three months and then every 3 months for the next 15 months. Removal of new surgically eligible BCCs (SEBs) will be done by primary skin care physicians (PSCPs) or at Study Centers. A Data Safety Monitoring Board (DSMB) will review unblinded results for an interim analysis when 20 subjects have completed 12 months of drug. This review will focus on adverse events and efficacy results. Subjects will be monitored for the development of new SEBs after they discontinue study treatment. At the end of the 18 months, given that the observed adverse events are minimal, patients on placebo will be offered the opportunity to take GDC-0449 for 18 months in an open label continuation, followed by six months observation, and patients on GDC449 will be monitored for the next 24 months for assessment of the duration of benefit after stopping the drug.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 41 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Phase II Multicenter Trial Evaluating the Efficacy and Safety of a Systemic Hedgehog Pathway Antagonist (GDC-0449) in Patients With Basal Cell Nevus Syndrome (BCNS)
Study Start Date : August 2009
Actual Primary Completion Date : January 2014
Actual Study Completion Date : January 2014

Arm Intervention/treatment
Placebo Comparator: Sugar pill
placebo pill by mouth once daily
Drug: GDC-0449
capsule, 150 mg, one pill daily, 18 months
Other Names:
  • Hedgehog Inhibitor
  • Hedgehog Antagonist

Experimental: GDC-0449
vismodegib 150MG by mouth once daily
Drug: GDC-0449
capsule, 150 mg, one pill daily, 18 months
Other Names:
  • Hedgehog Inhibitor
  • Hedgehog Antagonist

Primary Outcome Measures :
  1. OBJECTIVES Primary: - To evaluate the efficacy of GDC-0449 in reducing new surgically eligible BCCs in BCNS patients during month 3-18 of ingestion of 150 mg/day. - To evaluate the safety of this dose of GDC-0449 in these patients [ Time Frame: Monthly ]

Secondary Outcome Measures :
  1. Secondary: - To assess the effect of GDC-0449 in reducing the total number of BCCs of diameter 5mm or greater on the upper back. - To determine the duration of any anti-BCC effect of GDC-0449 after administration of the drug is stopped. [ Time Frame: Effacacy will assessed at 1 year and duration of anti-BCC effect will be assessed 6 months after study med is stopped. ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   35 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

The subject:

  • has had diagnosed at least 10 SEB (of diameter 3 mm diameter or greater on the nose or periorbital skin, 5 mm or greater elsewhere on the face, or 9 mm or greater on non-facial areas excluding the skin below the knees) during the two years before study entry, as documented histologically in physicians' records and/or diagnosed clinically by a Study Investigator at baseline.
  • meet diagnostic criteria for basal cell nevus syndrome
  • is willing to abstain from application of non-study topical medications to the skin for the duration of the study, including prescription and over the counter preparations. Subjects will be encouraged to use sunscreen (SPF 15) at least once daily on all exposed skin sites.
  • is willing to forego treatment of BCCs unless the BCCs are documented by Study Investigators, preferably on two separate visits, except when the PSCP believes that delay in treatment potentially might compromise the health of the subject.
  • has normal laboratory tests as defined by the following: Normal hematopoietic capacity, Normal hepatic function: AST and ALT greater than or equal to 2x the upper limit of normal (ULN) Total bilirubin within normal range 0.20 mg/dl to 1.50 mg/dl or within 3x ULN for patients with Gilbert's disease Normal renal function: normal serum creatinine or measured creatinine clearance less than 50 mL/minute. Fasting cholesterol greater than or equal to 220 untreated
  • be willing to not donate blood or semen for three months following discontinuation of Study medications.
  • is willing to avoid pregnancy in his partner as defined by the following: Male subject is willing to use a latex condom during the study and for 3 months after the last dose during sexual contact with a female of childbearing potential, even if he has had a successful vasectomy. His partner must also use a form of birth control

Exclusion Criteria:

The subject:

  • has used topical or systemic therapies that might interfere with the evaluation of the study medication during the study. Specifically these include the use of: (i) glucocorticoids to more than 5% of the skin (ii) retinoids systemically or topically to more than 5% of the skin during the six months prior to study entry; (iii) alpha-hydroxy acids to more than 5% of the skin during the six months prior to study entry (iv) 5-fluorouracil or imiquimod systemically or topically to the skin above the knees during the six months prior to study entry. (v) treatment with systemic chemotherapy within one year prior to starting study medication.
  • has a history of hypersensitivity to any of the ingredients in the study medication formulations.
  • is unable to return for follow-up visits and tests.
  • has uncontrolled systemic disease, including known HIV positive patients.
  • has history of congestive heart failure.
  • has uncontrolled hypocalcemia, hypomagnesemia, or hypokalemia
  • has clinically important history of liver disease, including viral or hepatitis, current alcohol abuse, or cirrhosis.
  • has any condition or situation which in the Investigator's opinion may put the subject at significant risk, could confound the study results, or could interfere significantly with the subject's participation in the study.
  • has a history of invasive cancer within the past five years excluding non-melanoma skin cancer, Stage I cervical cancer, ductal carcinoma in situ of the breast, or CLL Stage 0.
  • has current, recent (within 4 weeks of Day 1), or planned participation in an experimental drug study while enrolled in this study.
  • is a female who is pregnant, plans to ever to become pregnant, capable of becoming pregnant or is breast feeding.
  • is a male who is unwilling or unable to comply with pregnancy prevention measures.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00957229

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United States, California
Children's Hospital Oakland Research Institiute
Oakland, California, United States, 94609
United States, New York
Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center
New York, New York, United States, 10032
Sponsors and Collaborators
UCSF Benioff Children's Hospital Oakland
Genentech, Inc.
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Principal Investigator: Ervin Epstein, MD UCSF Benioff Children's Hospital Oakland
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: UCSF Benioff Children's Hospital Oakland Identifier: NCT00957229    
Other Study ID Numbers: 2009-026
SHH-4685s ( Other Grant/Funding Number: Genentech )
First Posted: August 12, 2009    Key Record Dates
Last Update Posted: June 6, 2016
Last Verified: January 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Additional relevant MeSH terms:
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Nevus, Pigmented
Basal Cell Nevus Syndrome
Pathologic Processes
Nevi and Melanomas
Neoplasms by Histologic Type
Odontogenic Cysts
Jaw Cysts
Bone Cysts
Carcinoma, Basal Cell
Neoplasms, Glandular and Epithelial
Neoplasms, Basal Cell
Neoplastic Syndromes, Hereditary
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Jaw Diseases
Stomatognathic Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn